CAMBRIDGE, Mass.--(BUSINESS WIRE)--Proclara Biosciences, a biotechnology company developing novel therapies that utilize the general amyloid interaction motif (GAIM) to treat disorders of protein misfolding, today announced the initiation of a Phase 1a clinical trial of NPT189. This next-generation molecule is in development for the treatment of systemic amyloidoses, which are a family of rare diseases that include amyloid light chain (AL) amyloidosis and hereditary transthyretin amyloidosis (ATTR). The objective of the trial is to evaluate the safety and tolerability of NPT189 in healthy volunteers.
Proclara also announced that it has completed enrollment in its ongoing Phase 1b clinical trial of NPT088 for the treatment of Alzheimer’s disease. This randomized, double-blind, placebo-controlled study is designed to evaluate the safety and tolerability of NPT088 and its effect on Alzheimer’s disease pathology as assessed by amyloid-β and tau PET imaging in patients with mild-to-moderate Alzheimer’s disease. Topline data from this study is expected in the first quarter of 2019.
“We are pleased to announce the expansion of our clinical development pipeline into orphan systemic diseases with NPT189, the second asset to emerge from our GAIM-based platform,” said Suzanne Bruhn, Ph.D., president and chief executive officer of Proclara. “This milestone, together with the completion of enrollment in our Phase 1b study of NPT088, marks an important step towards our goal of leveraging our unique technology to address a range of diseases caused by protein misfolding.”
“Our team is eager to evaluate NPT189, a potentially important new therapeutic, in patients with systemic amyloidoses, for whom no approved disease-modifying treatments currently exist,” said David Michelson, M.D., chief medical officer of Proclara. “We also look forward to obtaining the preliminary results from our Phase 1b study of NPT088 in patients with Alzheimer’s disease, and to the further progression of these therapeutic candidates in clinical development.”
About Systemic Amyloidoses
Systemic amyloidoses are life threatening diseases caused by the systemic buildup of amyloid protein fibers in organs and nerves. Over time, this build-up results in dysfunction that leads to significant morbidity and increased mortality. Current treatments are limited and new, more efficacious options are badly needed. Systemic amyloidoses are classified according to the type of deposited misfolded protein. Amyloid light chain (AL) amyloidosis is caused by an accumulation of misfolded immunoglobulin protein resulting in vital organ dysfunction, with up to 4,000 new cases diagnosed in the U.S. each year.1 Hereditary transthyretin (ATTR) amyloidosis, which is caused by mutant transthyretin (TTR) amyloid deposits in multiple organs, causes significant morbidity and mortality within two to 15 years from symptom onset and affects approximately 50,000 people worldwide.2,3 Proclara is developing NPT189, which targets LC and TTR deposits, for the treatment of these two orphan amyloidoses.
About Alzheimer’s Disease
Alzheimer’s disease is the most common neurodegenerative disease and the leading cause of dementia. A progressive and irreversible brain disorder that impacts memory and thinking skills, Alzheimer’s often affects the ability to carry out basic daily activities. The disease is characterized by the buildup of misfolded protein aggregates in the brain, including plaques, made of aggregated β-amyloid; tangles, made of aggregated tau; and sometimes α-synuclein aggregates. These toxic aggregates cause brain dysfunction and eventually, nerve cell death. Alzheimer’s affects approximately 47 million victims worldwide, and that number is expected to grow to 132 million by 2050.4
About Proclara Biosciences
Proclara Biosciences is a biotechnology company advancing product candidates developed based on its proprietary GAIM technology, which is capable of simultaneously targeting multiple toxic misfolded proteins. The broad applicability of GAIM technology enables the company to target multiple protein misfolding diseases, including neurodegenerative diseases and systemic amyloidoses. Proclara has developed a pipeline of drug candidates that use GAIM to target the common amyloid protein conformation, dissociating and preventing the formation of misfolded protein assemblies, and blocking the cell-to-cell transmission of toxic aggregates. No other single molecule or drug candidate displays such broad potential therapeutic activity for mitigating pathologic mechanisms in protein misfolding diseases. The company has two unique GAIM fusion proteins in clinical development. NPT088 is being evaluated in a Phase 1b trial for Alzheimer’s disease; and NPT189 is currently in Phase 1a in a clinical development program targeting systemic amyloidoses.
1. Amyloidosis Research Consortium, Guidance for Industry: AL Amyloidosis – Developing Drugs for Treatment, 2016 (www.arci.org)
2. Ando et al., Orphanet J Rare Dis, 2013
3. Ruberg et al., Circulation, 2012
4. Alzheimer’s Disease International, World Alzheimer Report, 2016, (www.alz.co.uk)
For more information, please visit proclarabio.com.